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Acorda Therapeutics, Inc. is a biotechnology company whose mission is to develop and market therapies that restore neurological function and improve the lives of people with multiple sclerosis (MS), spinal cord injury (SCI) and other disorders of the nervous system. The Company's marketed products include AMPYRA® (dalfampridine) Extended Release Tablets, 10 mg. and ZANAFLEX CAPSULES® (tizanidine hydrochloride). AMPYRA® is a potassium channel blocker approved as a treatment to improve walking in patients with multiple sclerosis (MS). This was demonstrated by an improvement in walking speed. ZANAFLEX CAPSULES® is a short-acting drug for the management of spasticity. The Company's pipeline includes a number of products in development for the treatment, regeneration and repair of the spinal cord and brain.

AlloCure is a Boston-based biotechnology company developing AC607, an allogeneic mesenchymal stem cell therapy for the treatment acute kidney injury (AKI) with potential applications in other grievous illnesses. 

Building on the success of a phase 1 clinical trial, AlloCure has initiated ACT-AKI, a randomized, multicenter, double-blind, placebo-controlled phase 2 trial in cardiac surgery subjects with post-operative AKI. Currently, there are no available treatment options for patients with AKI other than supportive care. AC607 represents the first application of a cell therapy to alter the course of this life threatening condition.

Conatus Pharmaceuticals Inc., a privately-held drug development company, is actively engaged in the development of first-in-class medicines to treat liver disease.  Liver disease affects hundreds of million people worldwide and can be caused by many different injuries to the liver including Hepatitis C and other viral infections, obesity, alcohol or autoimmune diseases

Emricasan (IDN-6556) is a first-in-class  oral small molecule compound to target apoptosis and treat liver fibrosis through a pan-caspase mechanism and is poised to enter Phase 3 registration studies in early 2013.

Emricasan is to be indicated for the treatment of HCV associated liver fibrosis in patients post orthotopic liver transplantation (POLT) to delay the progression of liver fibrosis to cirrhosis, and the treatment of acute liver failure in patients with underlying liver cirrhosis (ACLF) to improve transplant-free survival and decrease the incidence of progression to multi-organ failure.  Both diseases are potentially orphan indications.

Emricasan has completed the initial regulatory carcinogenicity study in rasH2 mice with an outcome of no drug related tumors observed.

Conatus has a broad portfolio of 128 issued patents plus pending applications protecting compounds and six gene families related to apoptosis that comprise viable drug targets; with exclusivity of emricasan franchise to 2028 w/o extensions

Emricasan’s initial ACLF and POLT orphan indications combined represent a potential revenue opportunity of ~1.6 billion in peak sales in US and EU and the estimated market launch is: ACLF (Q3 2016), POLT (Q4 2017).

Conatus is seeking a global or regional partner to advance Emricisan to the market.

Dicerna Technology: Next Generation RNAi and RNAi Delivery

Dicerna is a leading developer of RNAi drugs targeting unmet needs in cancer. The company’s novel Dicer Substrate siRNA (DsiRNA) molecules and proprietary drug delivery technologies have the demonstrated ability to silence previously undruggable disease targets. Dicerna’s RNAi platform is comprised of two key components: Dicer Substrates (DsiRNAs), which are 25-30 asymmetric double stranded RNAs that mediate RNAi with extremely high potency, and EnCore lipid nanoparticles, which are optimized for tumor delivery of RNAi molecules. The DsiRNA platform is broadly covered by the recently issued US patent 8,084,599.

Dicerna DsiRNA and Delivery Technologies enable breakthrough cancer treatments based on previously undruggable targets

Robust in vivo anti-tumor efficacy has been achieved in multiple difficult-to-treat tumors using DsiRNA Technology against classic undruggable oncology targets, both within our own pipeline and with our partner Kyowa Kirin. The company’s most advanced program targets the MYC oncogene and is focused on Hepatocellular Carcinoma (HCC), where there still remains a significant need for new treatment options. The HCC program is available for partnering.

Recent accomplishments

·         The demonstration of profound tumor responses in multiple solid tumor xenograft models.

·         The recent announcements that our partner, Kyowa Kirin, has moved the initial oncology candidate into formal development, triggering a substantial success milestone and the achievement of the in vivo efficacy milestone for the second DsiRNA oncology candidate.

·         The characterization and optimization of our EnCore lipid nanoparticle (LNP) technology and its ability to deliver DsiRNA payloads into the cell cytoplasm and achieve efficient knockdown of targeted genes.

·         The issuance of the fundamental patent covering the DsiRNA platform.

At Dicerna we can rapidly generate DsiRNA inhibitors against any gene and optimize our EnCore delivery technology to deliver to specific tumor types or organs.  We have demonstrated these capabilities in our lead HCC program, available for partnering, and can use these capabilities to develop therapeutics against targets of interest to collaborators.  I hope you have the time to meet with Dicerna during BioNetwork West to discuss our technology and areas of possible mutual interest.

The Company is a biopharmaceutical company that is developing and commercializing innovative medicines to target unmet therapeutic needs in arthritis, pain and inflammatory diseases. The Company has two FDA approved products: DUEXIS® and RAYOS®.  DUEXIS®s is a proprietary tablet formulation containing a fixed-dose combination of ibuprofen and famotidine in a single pill.  DUEXIS is indicated for the relief of signs and symptoms of rheumatoid arthritis (“RA”) and osteoarthritis and to decrease the risk of developing upper gastrointestinal ulcers in patients who are taking ibuprofen for these indications. In June 2012, the Company began expanding its commercial organization and expects to almost double its original field force size by the end of the third quarter of 2012, to approximately 150 field sales representatives. Also in June 2012, the Company engaged Mallinckrodt LLC, the pharmaceutical business of Covidien plc, on a non-exclusive basis to co-promote DUEXIS in the U.S. and entered into an exclusive collaboration, license and supply agreement with Grünenthal S.A. for the potential commercialization of DUEXIS in Latin America.

The Company’s second product, RAYOS®, known as LODOTRA® outside the U.S., is a proprietary delayed release formulation of low-dose prednisone that is currently marketed in Europe by its distribution partner, Mundipharma International Corporation Limited (“Mundipharma”), for the treatment of moderate to severe, active RA in adults, particularly when accompanied by morning stiffness. In addition, the Company has granted to Mundipharma commercialization rights to LODOTRA in Asia and Latin America. On July 26, 2012, the FDA approved RAYOS for the treatment of a broad range of diseases, including RA, polymyalgia rheumatica (“PMR”), psoriatic arthritis (“PsA”), ankylosing spondylitis (“AS”), asthma and chronic obstructive pulmonary disease (“COPD”). The Company anticipates the launch of RAYOS in the United States in the fourth quarter of 2012. The Company’s strategy is to commercialize its products in the U.S. and to enter into licensing or additional distribution agreements for commercialization of its products outside the U.S.

ImmunGene is leveraging promising science to empower therapeutic antibodies with immune molecules.  These molecules are highly optimized for dramatic efficacy and a superior safety profile. 

Our technology allows us to genetically engineer cytotoxic cytokines to antibodies to selectively target disease causing cells and tissues while reducing the systemic toxicity of the cytokines.  ImmunGene has several antibody molecules genetically engineered with interferon for following indications:

Non-Hodgkins lymphoma

Leukemia

Multiple myeloma

Melanoma

Renal cancer

Prostate cancer

Breast Cancer